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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01633489




Registration number
NCT01633489
Ethics application status
Date submitted
29/06/2012
Date registered
4/07/2012
Date last updated
27/09/2024

Titles & IDs
Public title
Lysosomal Acid Lipase (LAL) Deficiency Registry
Scientific title
An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
Secondary ID [1] 0 0
ALX-LALD-501
Universal Trial Number (UTN)
Trial acronym
ALX-LALD-501
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lysosomal Acid Lipase Deficiency 0 0
Cholesterol Ester Storage Disease 0 0
Wolman Disease 0 0
Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 0 0
Acid Lipase Deficiency 0 0
LIPA Deficiency 0 0
LAL-Deficiency 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
LAL Deficiency patients - Patients are those with a diagnosis of LAL Deficiency (living and deceased), irrespective of treatment status or treatment choice.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Understanding of the variability, progression, identification and natural history of LAL Deficiency.
Timepoint [1] 0 0
Ongoing

Eligibility
Key inclusion criteria
Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee.

Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/05/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/08/2029
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- New Lambton Heights
Recruitment postcode(s) [1] 0 0
- New Lambton Heights
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
State/province [2] 0 0
California
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United States of America
State/province [3] 0 0
Florida
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United States of America
State/province [4] 0 0
Georgia
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United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New Jersey
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Virginia
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
Belgium
State/province [15] 0 0
Ghent
Country [16] 0 0
Brazil
State/province [16] 0 0
Campinas
Country [17] 0 0
Brazil
State/province [17] 0 0
Ribeirão Preto
Country [18] 0 0
Brazil
State/province [18] 0 0
São Paulo
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Canada
State/province [20] 0 0
Edmonton
Country [21] 0 0
Canada
State/province [21] 0 0
Halifax
Country [22] 0 0
Croatia
State/province [22] 0 0
Zagreb
Country [23] 0 0
Czechia
State/province [23] 0 0
Olomouc
Country [24] 0 0
Czechia
State/province [24] 0 0
Prague
Country [25] 0 0
Denmark
State/province [25] 0 0
Copenhagen
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France
State/province [26] 0 0
Bron
Country [27] 0 0
France
State/province [27] 0 0
Clermont-Ferrand
Country [28] 0 0
France
State/province [28] 0 0
La Tronche
Country [29] 0 0
France
State/province [29] 0 0
Lyon
Country [30] 0 0
France
State/province [30] 0 0
Nancy
Country [31] 0 0
France
State/province [31] 0 0
Nantes
Country [32] 0 0
France
State/province [32] 0 0
Nice
Country [33] 0 0
France
State/province [33] 0 0
Paris
Country [34] 0 0
Germany
State/province [34] 0 0
Berlin
Country [35] 0 0
Germany
State/province [35] 0 0
Essen
Country [36] 0 0
Germany
State/province [36] 0 0
Hochheim
Country [37] 0 0
Germany
State/province [37] 0 0
Munich
Country [38] 0 0
Greece
State/province [38] 0 0
Athens
Country [39] 0 0
Ireland
State/province [39] 0 0
Dublin
Country [40] 0 0
Israel
State/province [40] 0 0
Jerusalem
Country [41] 0 0
Israel
State/province [41] 0 0
Petah Tikva
Country [42] 0 0
Italy
State/province [42] 0 0
Genoa
Country [43] 0 0
Italy
State/province [43] 0 0
Genova
Country [44] 0 0
Italy
State/province [44] 0 0
Milano
Country [45] 0 0
Italy
State/province [45] 0 0
Turin
Country [46] 0 0
Mexico
State/province [46] 0 0
Aguascalientes
Country [47] 0 0
Mexico
State/province [47] 0 0
Mexico City
Country [48] 0 0
Mexico
State/province [48] 0 0
Zapopan
Country [49] 0 0
Netherlands
State/province [49] 0 0
Amsterdam
Country [50] 0 0
Poland
State/province [50] 0 0
Warsaw
Country [51] 0 0
Portugal
State/province [51] 0 0
Guimarães
Country [52] 0 0
Portugal
State/province [52] 0 0
Lisbon
Country [53] 0 0
Saudi Arabia
State/province [53] 0 0
Jeddah
Country [54] 0 0
Saudi Arabia
State/province [54] 0 0
Riyadh
Country [55] 0 0
Slovenia
State/province [55] 0 0
Ljubljana
Country [56] 0 0
Spain
State/province [56] 0 0
Albacete
Country [57] 0 0
Spain
State/province [57] 0 0
Barcelona
Country [58] 0 0
Spain
State/province [58] 0 0
Madrid
Country [59] 0 0
Spain
State/province [59] 0 0
Oviedo
Country [60] 0 0
Spain
State/province [60] 0 0
Valladolid
Country [61] 0 0
Spain
State/province [61] 0 0
Zaragoza
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Birmingham
Country [63] 0 0
United Kingdom
State/province [63] 0 0
Cambridge
Country [64] 0 0
United Kingdom
State/province [64] 0 0
London
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Manchester
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.
Trial website
https://clinicaltrials.gov/study/NCT01633489
Trial related presentations / publications
Soll D, Spira D, Hollstein T, Haberbosch L, Demuth I, Steinhagen-Thiessen E, Bobbert T, Spranger J, Kassner U. Clinical outcome of a patient with lysosomal acid lipase deficiency and first results after initiation of treatment with Sebelipase alfa: A case report. Mol Genet Metab Rep. 2019 Jun 18;20:100479. doi: 10.1016/j.ymgmr.2019.100479. eCollection 2019 Sep.
Public notes

Contacts
Principal investigator
Name 0 0
Alexion Pharmaceuticals
Address 0 0
Sponsor GmbH
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01633489